An Open-Label, Long-Term Safety and Efficacy Evaluation of Diazoxide Choline Extended-Release Tablets in Participants With Prader-Willi Syndrome With a Double-Blind, Placebo-Controlled, Randomized Withdrawal Period
Overview
- Phase
- Phase 3
- Intervention
- DCCR
- Conditions
- Prader-Willi Syndrome
- Sponsor
- Soleno Therapeutics, Inc.
- Enrollment
- 115
- Locations
- 28
- Primary Endpoint
- Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating the safety and efficacy of DCCR treatment.
Detailed Description
115 PWS participants who completed clinical study C601 will be enrolled into the OLE Period. All participants in the Open Label Extension (OLE) Period will receive open-label DCCR. The actual number of participants eligible to enroll in the double-blind, placebo-controlled, randomized withdrawal (RW) period will be limited to those participants taking DCCR in the OLE Period at the time of the RW Period Visit 1 (Baseline/Randomization Visit).The treatment groups in the C602 RW Period are those participants randomized to receive DCCR and those participants randomized to receive Placebo. Participants will be randomized in a 1:1 ratio (DCCR:Placebo).
Investigators
Eligibility Criteria
Inclusion Criteria
- •Successful completion of clinical study C601
- •Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (subjects, as appropriate)
- •OLE Period Key
Exclusion Criteria
- •Positive urine pregnancy test (in females of child-bearing potential) or females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 90 days after study participation
- •Any new disease, condition, or circumstance which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol (e.g., an anticipated change of care setting)
- •RW Period Key Inclusion Criteria:
- •Provide voluntary, written informed consent (parent\[s\] / legal guardian\[s\] of participant); provide voluntary, written assent (participants, as appropriate); this includes consent for randomization and potential treatment with placebo for up to 16 weeks
- •Currently participating in clinical study C602 and complete the OLE End of Treatment Visit procedures
- •RW Period Key Exclusion Criteria:
- •Positive urine pregnancy test (in females of child-bearing potential)
- •Females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation
Arms & Interventions
OLE DCCR
75 - 525 mg DCCR
Intervention: DCCR
RW DCCR
75 - 525 mg DCCR
Intervention: DCCR
RW Placebo
75 - 525 mg Placebo for DCCR
Intervention: Placebo for DCCR
Outcomes
Primary Outcomes
Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported
Time Frame: Baseline to end of OLE (up to 4 years)
Safety analyses will be conducted in all participants who receive at least one dose of DCCR. Adverse events will be described by type and level of severity.
Change from RW Period Baseline in HQ-CT Total Score
Time Frame: RW Period Baseline to Week 16
Hyperphagia-related behaviors will be assessed by the hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors. The HQ-CT consists of nine items with responses ranging from 0-4 (best to worst). Scores from 9 items will be summed for a possible total score range of 0-36.
Secondary Outcomes
- Change in Body Fat Mass(Baseline to end of OLE (up to 4 years))
- Clinical Global Impression of Improvement (CGI-I)(RW Period Week 16)
- Clinical Global Impression of Severity (CGI-S)(RW Period Week 16)
- Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported(through the end of the RW Period, 16 weeks)
- Change from Baseline in HQ-CT Total Score(Baseline to end of OLE (up to 4 years))